Syndax Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company engaged in the development of cancer therapies. Its product candidates include SNDX-5613 and SNDX-6352. The company was founded by Richard A. Heyman, Eckard Weber, Peter Ordentlich, Ronald M. Evans and Michael Downes on October 11, 2005 and is headquartered in Waltham, MA.
Clinical Trials
35
6 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
N/A
Active, not recruiting
5
14.3%
Completed
18
51.4%
Not yet recruiting
1
2.9%
Recruiting
4
11.4%
Terminated
5
14.3%
Unknown
1
2.9%
Withdrawn
1
2.9%
No approval data available
- Step Pharma has advanced its first-in-class oral CTPS1 inhibitor dencatistat into a phase 2 clinical trial for essential thrombocythaemia, a rare clonal blood disorder with limited treatment options. - The phase 2 VECTRA trial will evaluate safety, tolerability, and efficacy in up to 50 high-risk ET patients across sites in the US, EU, and UK, following positive phase 1b data. - The company also announced the appointment of Dr. Neil Gallagher, a veteran oncology drug developer, as Non-Executive Director to support dencatistat's advancement across three clinical programmes. - Step Pharma expects to progress toward phase 3 trial initiation in ET during 2028, with dencatistat also being evaluated in lymphoma and solid tumour programmes.
- Syndax announced acceptance of four Revuforj abstracts at ASCO 2026, including an oral presentation showing favorable outcomes in 21 patients who received the menin inhibitor as post-transplant maintenance therapy. - The company will present 12 abstracts at EHA 2026, featuring real-world evidence and combination therapy data demonstrating strong activity across multiple genetic subtypes of acute leukemia. - New pharmacokinetic data will highlight Revuforj's unique profile, including the ability to administer with gastric acid reducing agents and low-fat meals without compromising efficacy.
- Syndax Pharmaceuticals and the World Orphan Drug Alliance announced a collaboration to launch a Managed Access Program for Revuforj (revumenib) in regions where the first-in-class menin inhibitor is not commercially available. - The program will expand access to Revuforj in parts of Eurasia, Central and Southeast Europe, Israel, the Middle East and Turkey, Latin America, and Africa for patients with relapsed or refractory acute leukemia. - Revuforj is FDA-approved for treating relapsed or refractory acute leukemia with KMT2A translocations and acute myeloid leukemia with NPM1 mutations in adult and pediatric patients one year and older. - The initiative aims to bridge the access gap for patients with rare and life-threatening diseases while allowing physicians to gain experience with the medicine globally.
- The FDA approved Kura Oncology's once-daily pill Komzifti (ziftomenib) for treating relapsed/refractory acute myeloid leukemia with NPM1 mutations, affecting about 30% of AML cases. - Komzifti gains a competitive safety edge over Syndax's recently approved Revuforj by avoiding a boxed warning for cardiac side effects, carrying only a regular warning for QTc prolongation. - The drug will be priced at $48,500 for a one-month supply and is expected to generate $1.32 billion in annual sales by 2031 according to analyst projections.
- UK-based Charm Therapeutics secured $80 million in Series B funding to advance its next-generation menin inhibitor for acute myeloid leukemia into Phase I clinical trials starting Q1 2026. - The company's AI-driven DragonFold platform designed a menin inhibitor that maintains potency against all known clinical resistance mutations, addressing key limitations of first-generation therapies. - The funding round was co-led by New Enterprise Associates and SR One, with participation from NVIDIA, OrbiMed, F-Prime, and Khosla Ventures. - Charm's candidate is designed to be efficacious at low doses while avoiding cardiac arrhythmia and drug-drug interactions associated with existing menin inhibitors.
- The HDAC inhibitors market across seven major markets is expected to surge significantly by 2034, driven by innovation in cancer therapies and expanding applications beyond traditional hematologic malignancies. - Several next-generation HDAC inhibitors are advancing through clinical trials, including Abexinostat in Phase III for renal cell carcinoma and Quisinostat in Phase II for uveal melanoma. - Recent market entrants include DUVYZAT for Duchenne muscular dystrophy and HIYASTA for T-cell lymphomas, demonstrating the therapeutic potential of HDAC inhibitors beyond oncology applications. - The market faces challenges from toxicity issues and competition from newer targeted therapies, but combination approaches with immune checkpoint inhibitors and precision oncology trends are creating new growth opportunities.
- Syndax Pharmaceuticals reported $28.6 million in Revuforj net revenue for Q2 2025, representing a 43% increase over the first quarter despite approximately one-third of patients pausing treatment for stem cell transplants. - The FDA granted Priority Review to Syndax's supplemental New Drug Application for Revuforj in relapsed/refractory mutant NPM1 acute myeloid leukemia, with a PDUFA action date of October 25, 2025. - Niktimvo generated $36.2 million in net revenue during its first full quarter of launch, with Syndax recording $9.4 million in collaboration revenue from its 50% profit-sharing agreement with Incyte. - The company maintains $517.9 million in cash and investments, which management expects will fund operations to profitability alongside growing product revenues from both approved therapies.
- Incyte reported strong second quarter 2025 financial results with Jakafi net revenues of $764 million, representing 8% year-over-year growth driven by increased demand across all indications. - The company achieved two significant regulatory milestones with FDA approvals of Zynyz for squamous cell anal carcinoma and Monjuvi for follicular lymphoma, expanding treatment options for cancer patients. - Niktimvo, launched in Q1 2025, demonstrated continued strong commercial uptake with $13.6 million in sales, while Opzelura revenues grew 35% to $164 million driven by increased patient demand in both atopic dermatitis and vitiligo. - The company raised its 2025 revenue guidance for Jakafi and other oncology products, reflecting strong first-half performance and successful product launches.
- Syndax Pharmaceuticals' REVUFORJ (revumenib) became the first FDA-approved menin inhibitor in November 2024, targeting KMT2A-rearranged acute leukemia patients aged one year and older. - Multiple companies including Kura Oncology, Sumitomo Pharma, and Johnson & Johnson are advancing next-generation menin inhibitors through clinical trials, with ziftomenib receiving breakthrough therapy designation for NPM1-mutant AML. - The market faces challenges from highly specific patient populations defined by genetic mutations and potential clinical resistance mechanisms, but shows promise for expansion into solid tumors and combination therapies. - Strategic partnerships and AI-driven drug development platforms are accelerating innovation, with companies exploring applications beyond hematologic cancers into diabetes and solid tumor indications.
- A recent budget impact analysis demonstrates that revumenib (Revuforj) for relapsed/refractory acute leukemias with KMT2A translocation would be cost-neutral for health plans over three years. - The cost neutrality is primarily driven by revumenib's oral administration, which eliminates expensive infusion-related costs including chair time and administration fees. - The analysis highlights challenges in evaluating rare disease treatments affecting only 1.7 individuals per million patients, where traditional cost-effectiveness methods may not adequately capture therapeutic value. - Patient adherence to the oral therapy remains a critical variable that could influence long-term cost-effectiveness and clinical outcomes.
